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NEUROGENCELL Main field : Clinical & translational neurosciences Secondary domain: Molecular & cellular neurosciences The "NEUROGENCELL" team develops gene and cell therapy strategies for severe neurodegenerative diseases of adults and children: Amyotrophic lateral sclerosis, Huntington's, Spinocerebellar ataxias, Parkinson's and Rett syndrome. As well as paediatric overload diseases: genetic leukodystrophies and mucopolysaccharidoses. This research includes proof of concepts in animal models and translational steps towards clinical applications. The "NEUROGENCELL" team is also developing tools for the entry of therapeutic molecules into the brain (vectors that can be administered by the venous route, optogenetics, use of microglia as a therapeutic vector, use of ultrasound to improve the passage of vectors across the blood-brain barrier).

Major publications

  • Piguet F., Alves S., Cartier N. Clinical Gene Therapy for Neurodegenerative diseases : Past, present and Future. Human gene therapy, oct 2017.
  • Piguet F., de Montigny C., Vaucamps N., Reutenauer L., Eisenmann A., Puccio H. Rapid and complete reversal of sensory ataxia by gene therapy using a novel mouse model of Friedreich ataxia. Molecular Therapy, June 2018.
  • Orefice, N.S, Souchet B., Breaudeau J., Alves S., Piguet F., Collaud F., Ronzitti G., Tada S., Hantraye P., Mingozzi F., Ducongé F., Cartier N. Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain. Methods & Clinical Development Volume 14, P237-251, September 2019.
  • The cholesterol 24-hydroxylase activates autophagy and decreases mutant huntingtin build-up in a neuroblastoma culture model of Huntington’s disease.
  • Nóbrega C, Conceição A, Costa RG, Koppenol R, Sequeira RL, Nunes R, Carmo-Silva S, Marcelo A, Matos CA, Betuing S, Caboche J, Cartier N, Alves S. BMC Res Notes. 2020 Apr 10;13(1):210. doi: 10.1186/s13104-020-05053-x. PMID: 32276655
  • Piguet F., de Saint Denis T., Audouard E.; Beccaria K., André A., Wurtz G., Schatz R., Alves S., Sevin C., Zerah M and Cartier N. The challenge of gene therapy for neurological diseases: strategies and tools to achieve efficient delivery to the central nervous system. Human Gene Therapy. In Press 2020.
  • Audouard E., Rousselot L., Folcher M., Cartier N. and Piguet F. Optimized protocol for sub-cutaneous implantation of encapsulated cells device and evaluation of biocompatibility. Frontiers in Neurosciences, May 2021.
  • Audouard E, Valentin O, B Meha, N Cartier, C Sevin and Piguet F. Optimized gene therapy approach for metachromatic leukodystrophy by intravenous gene delivery. Frontiers in Biomaterials, June 2021.
  • Habbas K., Cakil O., Zambo B., Tabet R., Riet F., Dembele D., Mandel J-L, Hocquemiller M., Laufer R., Piguet F. and Moine H. AAV- delivered diacylglycerol kinase DGKk achieves long-term rescue of fragile X syndrome mouse model. EMBO Mol Med 2022 Apr 4
  • Hematopoietic stem cell transplantation chemotherapy causes microglia senescence and peripheral macrophage engraftment in the brain.
  • Sailor KA, Agoranos G, López-Manzaneda S, Tada S, Gillet-Legrand B, Guerinot C, Masson JB, Vestergaard CL, Bonner M, Gagnidze K, Veres G, Lledo PM, Cartier N. Nat Med. 2022 Mar;28(3):517-527. doi: 10.1038/s41591-022-01691-9. Epub 2022 Feb 21. PMID: 35190726


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